Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases

Jacques P. Tremblay; Xiao Xiao; Annemieke Aartsma-Rus; Carlos Barbas; Helen M. Blau; Adam J. Bogdanove; Kym Boycott; Serge Braun; Xandra O. Breakefield; Juan A. Bueren; Michael Buschmann; Barry J. Byrne; Michele Calos; Toni Cathomen; Jeffrey Chamberlain; Marinee Chuah; Kenneth Cornetta; Kay E. Davies; J. George Dickson; Philippe Duchateau; Terence R. Flotte; Daniel Gaudet; Charles A. Gersbach; Renald Gilbert; Joseph Glorioso; Roland W. Herzog; Katherine A. High; Wenlin Huang; Johnny Huard; J. Keith Joung; Depei Liu; Dexi Liu; Hanns Lochmüller; Lawrence Lustig; Jeffrey Martens; Bernard Massie; Fulvio Mavilio; Jerry R. Mendell; Amit Nathwani; Katherine Ponder; Matthew Porteus; Jack Puymirat; Jude Samulski; Shin'Ichi Takeda; Adrian Thrasher; Thierry Vandendriessche; Yuquan Wei; James M. Wilson; Steve D. Wilton; John H. Wolfe; Guangping Gao

doi:10.1038/mt.2013.4

Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases

Jacques P. Tremblay, Xiao Xiao, Annemieke Aartsma-Rus, Carlos Barbas, Helen M. Blau, Adam J. Bogdanove, Kym Boycott, Serge Braun, Xandra O. Breakefield, Juan A. Bueren, Michael Buschmann, Barry J. Byrne, Michele Calos, Toni Cathomen, Jeffrey Chamberlain, Marinee Chuah, Kenneth Cornetta, Kay E. Davies, J. George Dickson, Philippe DuchateauTerence R. Flotte, Daniel Gaudet, Charles A. Gersbach, Renald Gilbert, Joseph Glorioso, Roland W. Herzog, Katherine A. High, Wenlin Huang, Johnny Huard, J. Keith Joung, Depei Liu, Dexi Liu, Hanns Lochmüller, Lawrence Lustig, Jeffrey Martens, Bernard Massie, Fulvio Mavilio, Jerry R. Mendell, Amit Nathwani, Katherine Ponder, Matthew Porteus, Jack Puymirat, Jude Samulski, Shin'Ichi Takeda, Adrian Thrasher, Thierry Vandendriessche, Yuquan Wei, James M. Wilson, Steve D. Wilton, John H. Wolfe, Guangping Gao

Division of Hematology

Research output: Contribution to journal › Letter › peer-review

10 Scopus citations

Original language	English
Pages (from-to)	266-268
Number of pages	3
Journal	Molecular Therapy
Volume	21
Issue number	2
DOIs	https://doi.org/10.1038/mt.2013.4
State	Published - Feb 2013

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Tremblay, J. P., Xiao, X., Aartsma-Rus, A., Barbas, C., Blau, H. M., Bogdanove, A. J., Boycott, K., Braun, S., Breakefield, X. O., Bueren, J. A., Buschmann, M., Byrne, B. J., Calos, M., Cathomen, T., Chamberlain, J., Chuah, M., Cornetta, K., Davies, K. E., Dickson, J. G., ... Gao, G. (2013). Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases. Molecular Therapy, 21(2), 266-268. https://doi.org/10.1038/mt.2013.4

@article{b4e862b29bb142ec82c09526029897a0,

title = "Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases",

author = "Tremblay, {Jacques P.} and Xiao Xiao and Annemieke Aartsma-Rus and Carlos Barbas and Blau, {Helen M.} and Bogdanove, {Adam J.} and Kym Boycott and Serge Braun and Breakefield, {Xandra O.} and Bueren, {Juan A.} and Michael Buschmann and Byrne, {Barry J.} and Michele Calos and Toni Cathomen and Jeffrey Chamberlain and Marinee Chuah and Kenneth Cornetta and Davies, {Kay E.} and Dickson, {J. George} and Philippe Duchateau and Flotte, {Terence R.} and Daniel Gaudet and Gersbach, {Charles A.} and Renald Gilbert and Joseph Glorioso and Herzog, {Roland W.} and High, {Katherine A.} and Wenlin Huang and Johnny Huard and Joung, {J. Keith} and Depei Liu and Dexi Liu and Hanns Lochm{\"u}ller and Lawrence Lustig and Jeffrey Martens and Bernard Massie and Fulvio Mavilio and Mendell, {Jerry R.} and Amit Nathwani and Katherine Ponder and Matthew Porteus and Jack Puymirat and Jude Samulski and Shin'Ichi Takeda and Adrian Thrasher and Thierry Vandendriessche and Yuquan Wei and Wilson, {James M.} and Wilton, {Steve D.} and Wolfe, {John H.} and Guangping Gao",

note = "Funding Information: Over the past decade, gene therapy has been successfully used to treat several monogenic disorders, and it shows promise for treating diseases of more complex etiology. In addition, the recent development of induced pluripo tent stem cells now opens the possibility of transplanting genetically corrected autologous cells.1,2Very recently, the European Medicines Agency approved the first gene therapy treatment in the Western world.3The substantial progress over the preceding decades arguably portends the development of gene therapies for most monogenic diseases. Given this remarkable opportunity, we are proposing the creation of an International Gene Therapy Consortium for Monogenic Diseases. This consortium would facilitate coordination of the production and availability of a variety of vectors, oligonucleotides, and recombinant proteins—including zinc-finger nucleases and Tal effector nucleases—as well as support the development of suitable animal models, preclinical studies, and clinical trials. Financial resources should be developed so as to attract collaborations from the private sector to boost the development of a gene therapy industry, similar to the way the Apollo project to explore the moon stimulated growth of the space and computer industries in the 1960s. In this century, a similar concerted effort will be required to develop effective treatments and even cures of diseases here on Earth! A model for such a consortium can be found in the field of genomics. Advances in genomics have been rapid, owing in large part to the formation of international consortia such as the Human Genome and the ENCODE (Encyclopedia of DNA Elements) proj ects. These consortia have been awarded large budgets by various government agencies that have permitted intense collaboration among scientists as well as engagement of industry for the development of supporting technologies. The funding made available for these projects contrasts sharply with the relatively limited budgets that have been available for gene therapy research. Typically, most gene therapy researchers work as small teams on a specific disease with a relatively small budget. Moreover, the funding for gene therapy research tends to be piecemeal, with part coming from private foundations supported by patients, parents, and friends. Although these small groups can provide proof of concept for a gene therapy approach in cell and animal models, they generally lack the expertise and funding to efficiently translate their strategies to a clinical trial.",

year = "2013",

month = feb,

doi = "10.1038/mt.2013.4",

language = "English",

volume = "21",

pages = "266--268",

journal = "Molecular Therapy",

issn = "1525-0016",

number = "2",

}

Tremblay, JP, Xiao, X, Aartsma-Rus, A, Barbas, C, Blau, HM, Bogdanove, AJ, Boycott, K, Braun, S, Breakefield, XO, Bueren, JA, Buschmann, M, Byrne, BJ, Calos, M, Cathomen, T, Chamberlain, J, Chuah, M, Cornetta, K, Davies, KE, Dickson, JG, Duchateau, P, Flotte, TR, Gaudet, D, Gersbach, CA, Gilbert, R, Glorioso, J, Herzog, RW, High, KA, Huang, W, Huard, J, Joung, JK, Liu, D, Liu, D, Lochmüller, H, Lustig, L, Martens, J, Massie, B, Mavilio, F, Mendell, JR, Nathwani, A, Ponder, K, Porteus, M, Puymirat, J, Samulski, J, Takeda, SI, Thrasher, A, Vandendriessche, T, Wei, Y, Wilson, JM, Wilton, SD, Wolfe, JH & Gao, G 2013, 'Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases', Molecular Therapy, vol. 21, no. 2, pp. 266-268. https://doi.org/10.1038/mt.2013.4

TY - JOUR

T1 - Translating the genomics revolution

T2 - The need for an international gene therapy consortium for monogenic diseases

AU - Tremblay, Jacques P.

AU - Xiao, Xiao

AU - Aartsma-Rus, Annemieke

AU - Barbas, Carlos

AU - Blau, Helen M.

AU - Bogdanove, Adam J.

AU - Boycott, Kym

AU - Braun, Serge

AU - Breakefield, Xandra O.

AU - Bueren, Juan A.

AU - Buschmann, Michael

AU - Byrne, Barry J.

AU - Calos, Michele

AU - Cathomen, Toni

AU - Chamberlain, Jeffrey

AU - Chuah, Marinee

AU - Cornetta, Kenneth

AU - Davies, Kay E.

AU - Dickson, J. George

AU - Duchateau, Philippe

AU - Flotte, Terence R.

AU - Gaudet, Daniel

AU - Gersbach, Charles A.

AU - Gilbert, Renald

AU - Glorioso, Joseph

AU - Herzog, Roland W.

AU - High, Katherine A.

AU - Huang, Wenlin

AU - Huard, Johnny

AU - Joung, J. Keith

AU - Liu, Depei

AU - Liu, Dexi

AU - Lochmüller, Hanns

AU - Lustig, Lawrence

AU - Martens, Jeffrey

AU - Massie, Bernard

AU - Mavilio, Fulvio

AU - Mendell, Jerry R.

AU - Nathwani, Amit

AU - Ponder, Katherine

AU - Porteus, Matthew

AU - Puymirat, Jack

AU - Samulski, Jude

AU - Takeda, Shin'Ichi

AU - Thrasher, Adrian

AU - Vandendriessche, Thierry

AU - Wei, Yuquan

AU - Wilson, James M.

AU - Wilton, Steve D.

AU - Wolfe, John H.

AU - Gao, Guangping

N1 - Funding Information: Over the past decade, gene therapy has been successfully used to treat several monogenic disorders, and it shows promise for treating diseases of more complex etiology. In addition, the recent development of induced pluripo tent stem cells now opens the possibility of transplanting genetically corrected autologous cells.1,2Very recently, the European Medicines Agency approved the first gene therapy treatment in the Western world.3The substantial progress over the preceding decades arguably portends the development of gene therapies for most monogenic diseases. Given this remarkable opportunity, we are proposing the creation of an International Gene Therapy Consortium for Monogenic Diseases. This consortium would facilitate coordination of the production and availability of a variety of vectors, oligonucleotides, and recombinant proteins—including zinc-finger nucleases and Tal effector nucleases—as well as support the development of suitable animal models, preclinical studies, and clinical trials. Financial resources should be developed so as to attract collaborations from the private sector to boost the development of a gene therapy industry, similar to the way the Apollo project to explore the moon stimulated growth of the space and computer industries in the 1960s. In this century, a similar concerted effort will be required to develop effective treatments and even cures of diseases here on Earth! A model for such a consortium can be found in the field of genomics. Advances in genomics have been rapid, owing in large part to the formation of international consortia such as the Human Genome and the ENCODE (Encyclopedia of DNA Elements) proj ects. These consortia have been awarded large budgets by various government agencies that have permitted intense collaboration among scientists as well as engagement of industry for the development of supporting technologies. The funding made available for these projects contrasts sharply with the relatively limited budgets that have been available for gene therapy research. Typically, most gene therapy researchers work as small teams on a specific disease with a relatively small budget. Moreover, the funding for gene therapy research tends to be piecemeal, with part coming from private foundations supported by patients, parents, and friends. Although these small groups can provide proof of concept for a gene therapy approach in cell and animal models, they generally lack the expertise and funding to efficiently translate their strategies to a clinical trial.

PY - 2013/2

Y1 - 2013/2

UR - http://www.scopus.com/inward/record.url?scp=84873333610&partnerID=8YFLogxK

U2 - 10.1038/mt.2013.4

DO - 10.1038/mt.2013.4

M3 - Letter

C2 - 23369965

AN - SCOPUS:84873333610

SN - 1525-0016

VL - 21

SP - 266

EP - 268

JO - Molecular Therapy

JF - Molecular Therapy

IS - 2

ER -

Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases

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